Positive Phase III results for valoctocogene roxaparvovec in the treatment of severe haemophilia A
In the GENEr8-1 study (n=134), a single dose of valoctocogene roxaparvovec reduced annualised bleeding rate from a mean of 4.8 episodes per year at baseline to 0.8 per year, after a mean follow-up of 71.6 weeks (p<0.0001).
Source:
Biospace Inc.
SPS commentary:
These results were from a pre-specified group of prior participants in a non-interventional baseline observational study (n=112), conducted to assess bleeding episodes, Factor VIII use and health-related quality of life while receiving Factor VIII prophylaxis prior to rolling over to receive treatment in the GENEr8-1 study. Mean annualised Factor VIII in this population was reduced from 135.9 to 2.0 infusions per year after receiving valoctocogene roxaparvovec (p<0.0001).